Table 6.
ATMP candidates in the industrial pipeline in phase III/IV clinical trial.
| ATMP candidate | Clinical trial identifier | Sponsor | Indication | Study State |
|---|---|---|---|---|
| Autologous CD133+ bone marrow stem cells | NCT00950274 | Miltenyi Biotec GmbH | Chronic ischemic coronary artery disease | Terminated (slow recruitment) |
| Skeletal muscle derived cells | 2014–001656-34 | Innovacell Biotechnologie AG | Stress urinary incontinence | Completed |
| Tumor site allografts of healthy endothelial cells embedded in polymer matrix | Unknown | Shire (Pervasis) | Treatment/ Prevention of metastatic cancer |
Unknown |
| Bone marrow derived mononuclear cells | NCT01285297 | Cardiogenesis | Transmyocardial revascularisation | Completed |
| C-CURE (bone marrow derived cardiopoietic cells) | NCT01768702 | Celyad (formerly named Cardio3 BioSciences) | Ischaemic heart failure | Completed |
| Generx (FGF-4 gene therapy) | NCT02928094 | Angionetics Inc. | Coronary artery disease | Completed |
| Riferminogene pecaplasmid (Gene therapy) | NCT00566657 | Sanofi | Critical limb ischemia | Completed (failure to detect efficacy) |
| Cerepro (cancer vaccine) | EUDRACT2004-000464–28. | Ark Therapeutics | Operable high-grade glioma | Completed; MAA withdrawn (unable to demonstrate a clinically meaningful benefit) |
| Autologous chondrocytes | EUDRACT2016-002817–22 | TETEC Tissue Engineering Technologies – AG | Cartilage damage | Active |
| Keratinocytes | EUDRACT2012-003286–18 | Smith & Nephew | Chronic leg ulcer | Terminated (failure to detect efficacy) |
| Autologous dendritic cells | EUDRACT2012-000871–17 | Argos Therapeutics, Inc. | Renal cell carcinoma stage IV | Active |
| Autologous dendritic cells | NCT02111577 | Sotio a.s. | Metastatic castration resistant prostate cancer | Active |
| Cancer vaccine | NCT01383148 | Transgene | Non small cell lung cancer | Terminated (reason unclear) |
| Pexa-Vec (Cancer vaccine) | NCT02562755 | SillaJen, Inc. | Hepatocellular Carcinoma | Recruiting |
| AVXS-101 (Gene Therapy) | NCT03461289 | AveXis, Inc. | Spinal Muscle Atrophy | Recruiting |
| CER-001 (Gene Therapy) | NCT02697136 | Cerenis Therapeutics, SA | Primary Hypoalphalipoproteinemia | Recruiting |
| Autologous fat enhanced with regenerative cells | NCT00616135 | Cytori Therapeutics | Cosmetic breast deformities | Completed |
| AMG0001 (Gene Therapy) | NCT02144610 | AnGes USA, Inc. | Critical limb ischemia | Terminated (strategy amendment) |
| Autologous Muscle Derived Cells | NCT01893138 | Cook MyoSite|Cook Group Incorporated | Female Urinary Sphincter Repair | Active, not recruiting |
| Cancer vaccine | NCT01817738 | CureVac AG | Prostate cancer | Terminated |
| Valoctocogene Roxaparvovec (Gene Therapy) | NCT03392974 | BioMarin Pharmaceutical | Hemophilia A | Recruiting |
| AAVrh10-h.SGSH Gene Therapy | NCT03612869 | LYSOGENE | Mucopoly-saccharidosis | Not yet recruiting |
| Bone marrow stem cells | NCT00462774 | Miltenyi Biotec GmbH | Ischaemic heart failure | Completed |
| Progenitor Cells | NCT00279175 | Eli Lilly and Company | Acute Myocardial Infarction | Completed |
| Cancer vaccine | NCT00676507 | NovaRx Corporation | Non-small Cell Lung Cancer | Completed |
| GSK2696274 (Gene Therapy) | NCT03392987 | GlaxoSmithKline | Metachromatic Leukodystrophy | Recruiting |
| Mesenchymal Stem Cells | NCT00366145 | Osiris Therapeutics | Acute Graft Versus Host Disease | Completed |
| NiCord® cord blood stem cells | NCT02730299 | Gamida Cell ltd | Hematologic malignancies | Recruiting |
| TG4010 (Cancer vaccine) | NCT00415818 | Transgene | Non-Small Cell Lung Cancer | Completed |
| DCVax®-L (Cancer vaccine) | NCT00045968 | Northwest Biotherapeutics | Glioblastoma | Unknown status |
| AAV2-REP1(Gene Therapy) | NCT03496012 | Nightstar Therapeutics | Choroideremia | Recruiting |